WO2000075279A3 - Nucleotide sequences for gene regulation and methods of use thereof - Google Patents

Nucleotide sequences for gene regulation and methods of use thereof Download PDF

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Publication number
WO2000075279A3
WO2000075279A3 PCT/US2000/015728 US0015728W WO0075279A3 WO 2000075279 A3 WO2000075279 A3 WO 2000075279A3 US 0015728 W US0015728 W US 0015728W WO 0075279 A3 WO0075279 A3 WO 0075279A3
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WO
WIPO (PCT)
Prior art keywords
methods
interest
nucleic acid
acid sequences
liver
Prior art date
Application number
PCT/US2000/015728
Other languages
French (fr)
Other versions
WO2000075279A2 (en
Inventor
Kotoku Kurachi
Sumiko Kurachi
Original Assignee
Univ Michigan
Kotoku Kurachi
Sumiko Kurachi
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Michigan, Kotoku Kurachi, Sumiko Kurachi filed Critical Univ Michigan
Priority to EP00938213A priority Critical patent/EP1581614A4/en
Priority to JP2001502546A priority patent/JP2003527081A/en
Priority to AU53286/00A priority patent/AU5328600A/en
Publication of WO2000075279A2 publication Critical patent/WO2000075279A2/en
Publication of WO2000075279A3 publication Critical patent/WO2000075279A3/en

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/644—Coagulation factor IXa (3.4.21.22)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A61P19/10—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/647—Blood coagulation factors not provided for in a preceding group or according to more than one of the proceeding groups
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)

Abstract

The invention provides nucleic acid sequences which regulate expression of a nucleotide sequence of interest. In particular, the invention provides nucleic acid sequences which regulate expression of a nucleotide sequence of interest in an age-related manner and/or in a liver-specific manner. The invention further provides methods of using the regulatory nucleic acid sequences provided herein for age-related and/or liver-specific expression of nucleotides sequences of interest. The invention also provides host cells and transgenic non-human animals which harbor the regulatory nucleic acid sequences of the invention. The compositions and methods of the invention are useful in regulating expression of a nucleotide sequence of interest in an age-related and/or liver-specific manner.
PCT/US2000/015728 1999-06-09 2000-06-06 Nucleotide sequences for gene regulation and methods of use thereof WO2000075279A2 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
EP00938213A EP1581614A4 (en) 1999-06-09 2000-06-06 Nucleotide sequences for gene regulation and methods of use thereof
JP2001502546A JP2003527081A (en) 1999-06-09 2000-06-06 Nucleic acid sequences for gene regulation and methods of use
AU53286/00A AU5328600A (en) 1999-06-09 2000-06-06 Nucleotide sequences for gene regulation and methods of use thereof

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US09/328,925 1999-06-09
US09/328,925 US6610906B1 (en) 1999-06-09 1999-06-09 Nucleotide sequences for gene regulation and methods of use thereof

Publications (2)

Publication Number Publication Date
WO2000075279A2 WO2000075279A2 (en) 2000-12-14
WO2000075279A3 true WO2000075279A3 (en) 2005-09-22

Family

ID=23283070

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2000/015728 WO2000075279A2 (en) 1999-06-09 2000-06-06 Nucleotide sequences for gene regulation and methods of use thereof

Country Status (6)

Country Link
US (2) US6610906B1 (en)
EP (1) EP1581614A4 (en)
JP (1) JP2003527081A (en)
KR (1) KR100530298B1 (en)
AU (1) AU5328600A (en)
WO (1) WO2000075279A2 (en)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6262249B1 (en) * 1998-06-23 2001-07-17 Chiron Corporation Pancreatic cancer genes
US7351813B2 (en) * 2000-06-20 2008-04-01 The Board Of Trustees Of The Leland Stanford Junior University Liver-specific gene expression cassettes, and methods of use
US20040088744A1 (en) * 2000-08-14 2004-05-06 Alan Attie Fatty liver disease resistant bovines
WO2002083921A2 (en) 2001-04-10 2002-10-24 Agensys, Inc. Nuleic acids and corresponding proteins useful in the detection and treatment of various cancers
JP2011502515A (en) * 2007-11-09 2011-01-27 アイシス ファーマシューティカルズ インコーポレイティッド Regulation of factor 9 expression
KR101190591B1 (en) 2008-02-14 2012-10-15 재단법인 목암생명공학연구소 Expression vector suitable for expression of a coding sequence for gene therapy
TWI791433B (en) 2016-04-15 2023-02-11 賓州大學委員會 Gene therapy for treating hemophilia a
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
US11306329B2 (en) 2018-02-19 2022-04-19 City Of Hope Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof
TW202140791A (en) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 Methods of treating phenylketonuria

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1998041240A1 (en) * 1997-03-14 1998-09-24 The Children's Hospital Of Philadelphia Methods and compositions for use in gene therapy for treatment of hemophilia
US5814716A (en) * 1989-08-09 1998-09-29 Transgene S.A. Cell lines from a transgenic mouse which express biologically active IX factor

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4965188A (en) 1986-08-22 1990-10-23 Cetus Corporation Process for amplifying, detecting, and/or cloning nucleic acid sequences using a thermostable enzyme
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US5545806A (en) 1990-08-29 1996-08-13 Genpharm International, Inc. Ransgenic non-human animals for producing heterologous antibodies
US5625126A (en) 1990-08-29 1997-04-29 Genpharm International, Inc. Transgenic non-human animals for producing heterologous antibodies
KR100272077B1 (en) 1990-08-29 2000-11-15 젠팜인터내셔날,인코포레이티드 Transgenic non-human animals capable of producing heterologous antibodies
US5591601A (en) 1993-05-14 1997-01-07 Ohio University Edison Animal Biotechnology Institute DNA polymerase gene expression system utilizing an RNA polymerase co-delivered with the gene expression vector system

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5814716A (en) * 1989-08-09 1998-09-29 Transgene S.A. Cell lines from a transgenic mouse which express biologically active IX factor
WO1998041240A1 (en) * 1997-03-14 1998-09-24 The Children's Hospital Of Philadelphia Methods and compositions for use in gene therapy for treatment of hemophilia

Non-Patent Citations (11)

* Cited by examiner, † Cited by third party
Title
JALLAT S ET AL: "Characterization of recombinant human factor IX expressed in transgenic mice and in derived transimmortalized hepatic cell lines", EMBO JOURNAL, OXFORD UNIVERSITY PRESS, SURREY, GB, vol. 9, no. 10, 1990, pages 3295 - 3301, XP002044830, ISSN: 0261-4189 *
KURACHI K ET AL: "Genetic and molecular mechanisms of age regulation (homeostasis) of blood coagulation", IUBMB LIFE, TAYLOR AND FRANCIS, LONDON,, GB, vol. 49, no. 3, March 2000 (2000-03-01), pages 189 - 196, XP001117892, ISSN: 1521-6543 *
KURACHI K ET AL: "Regulatory mechanism of the factor IX gene", THROMBOSIS AND HAEMOSTASIS, STUTTGART, DE, vol. 73, no. 3, March 1995 (1995-03-01), pages 333 - 339, XP000938914, ISSN: 0340-6245 *
KURACHI S ET AL: "Genetic mechanisms of age regulation of human blood coagulation factor IX.", SCIENCE (WASHINGTON D C), vol. 285, no. 5428, 30 July 1999 (1999-07-30), pages 739 - 743, XP002218779, ISSN: 0036-8075 *
KURACHI S ET AL: "Molecular mechanisms of homeostasis in blood coagulation: Age-associated regulation of the human factor IX gene.", BLOOD, vol. 92, no. 10 SUPPL. 1 PART 1-2, 15 November 1998 (1998-11-15), 40th Annual Meeting of the American Society of Hematology;Miami Beach, Florida, USA; December 4-8, 1998, pages 709A, XP002218778, ISSN: 0006-4971 *
KURACHI S ET AL: "Role of intron I in expression of the human factor IX gene", JOURNAL OF BIOLOGICAL CHEMISTRY, AMERICAN SOCIETY OF BIOLOGICAL CHEMISTS, BALTIMORE, MD, US, vol. 270, no. 10, 10 March 1995 (1995-03-10), pages 5276 - 5281, XP002055415, ISSN: 0021-9258 *
MIAO C H ET AL: "Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo bur not in vitro", MOLECULAR THERAPY, ACADEMIC PRESS, SAN DIEGO, CA,, US, vol. 1, no. 6, June 2000 (2000-06-01), pages 522 - 532, XP001063991, ISSN: 1525-0016 *
MIAO CAROL H ET AL: "Transcriptional regulation of the gene coding for human protein C.", JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 271, no. 16, 1996, pages 9587 - 9594, XP002234314, ISSN: 0021-9258 *
SALIER J-P ET AL: "Functional characterization of the 5'-regulatory region of human factor IX gene", JOURNAL OF BIOLOGICAL CHEMISTRY, AMERICAN SOCIETY OF BIOLOGICAL CHEMISTS, BALTIMORE, MD, US, vol. 265, no. 12, 25 April 1990 (1990-04-25), pages 7062 - 7068, XP002116374, ISSN: 0021-9258 *
See also references of EP1581614A4 *
ZHANG KEZHONG ET AL: "Genetic mechanisms of age regulation of protein C and blood coagulation.", JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 277, no. 6, 8 February 2002 (2002-02-08), February 8, 2002, pages 4532 - 4540, XP002234315, ISSN: 0021-9258 *

Also Published As

Publication number Publication date
AU5328600A (en) 2000-12-28
EP1581614A2 (en) 2005-10-05
KR20020038589A (en) 2002-05-23
US6610906B1 (en) 2003-08-26
WO2000075279A2 (en) 2000-12-14
EP1581614A4 (en) 2005-10-05
US7341871B2 (en) 2008-03-11
US20060031956A1 (en) 2006-02-09
JP2003527081A (en) 2003-09-16
KR100530298B1 (en) 2005-11-22

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